Acadia Pharmaceuticals Hosts Inaugural R&D Day Showcasing Pipeline and Long-Term Value Drivers

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) will host its inaugural 2025 R&D Day today to highlight its key pipeline programs and their potential to drive long-term growth.

Across nine disclosed programs, the Company anticipates initiating seven Phase 2 or Phase 3 studies during 2025-2026. Acadia further anticipates reporting five Phase 2 or Phase 3 study readouts during 2025-2027—underscoring the breadth of its pipeline and the momentum behind its R&D strategy.

ANTICIPATED PIPELINE MILESTONES

• Q3 2025 - Phase 2 study initiation for ACP-204 in Lewy Body Dementia Psychosis (LBDP)
• Q3 2025 - Phase 3 study initiation in Japan for trofinetide in Rett syndrome
• Early Q4 2025 - Topline results for the COMPASS PWS Phase 3 study of ACP-101 in Prader-Willi syndrome (PWS) following the recent completion of enrollment
• Q4 2025 - Phase 2 study initiation for ACP-211 in major depressive disorder (MDD)
• Q4 2025 - First in human study of ACP-271 in healthy volunteers
• Q1 2026 - Potential approval from European Medicines Agency for trofinetide
• Mid-2026 - Topline results from ACP-204 Phase 2 study in Alzheimer’s disease psychosis (ADP)
• 2026 - Phase 2 study initiation for ACP-711 in Essential Tremor (ET)

“Acadia is building from a position of strength, with two commercial franchises on track to deliver over $1 billion in net sales this year,” said Catherine Owen Adams, Chief Executive Officer. “As we advance our pipeline, the five molecules we showcase today represent a multibillion-dollar incremental revenue opportunity. They also underscore our unwavering commitment to bold science and our focus on delivering transformative therapies for patients with serious and underserved neurological and rare diseases. We believe the opportunity ahead is extraordinary—and we are confident in our ability to execute with discipline and purpose as we work to build a biotech powerhouse.”

“At Acadia, we’re building a strong, sustainable pipeline focused on progressing the most promising science with the goal of meaningfully improving patient care,” said Elizabeth H.Z. Thompson, Ph.D., Executive Vice President, Head of R&D. “Our pipeline is moving forward with real momentum. Between now and the end of 2026, we plan to initiate seven Phase 2 or 3 studies, with five key readouts anticipated by the end of 2027. We remain deeply committed to advancing care in neurological and rare diseases, leveraging our development expertise and commercial strength to bring meaningful innovation to patients.”

The Company will be discussing the following pipeline programs today:

ACP-101 (intranasal carbetocin)

• A long-acting analogue of human oxytocin for the potential treatment of PWS

ACP-204

• A new, highly selective, potent inverse agonist of 5-HT2A that builds upon lessons learned from pimavanserin for the potential treatment of ADP and LBDP

ACP-211

• An orally administered, selectively deuterated form of R-Norketamine for the potential treatment of MDD

ACP-711

• A selective GABA_A-α3 modulator targeting the potential treatment of ET

ACP-271

• A GPR88 agonist for the potential treatment of Tardive Dyskinesia and Huntington’s Disease

To register for the live webcast, please click here. A replay of Acadia’s R&D Day will be available on the company’s website, Acadia.com, under the investors section for approximately 3 months following the event.

About Acadia Pharmaceuticals

Acadia is advancing breakthroughs in neurological and rare diseases to elevate life. Since our founding we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only approved drug in the United States and Canada for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Prader-Willi syndrome, Alzheimer’s disease psychosis and multiple other programs targeting neuroscience and neuro-rare diseases. For more information, visit us at Acadia.com and follow us on LinkedIn and X.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential,” “continue,” “opportunity,” “goal” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) our business strategy, objectives and opportunities, including the timing for and clinical and market potential of our pipeline assets, and potential for enhanced shareholder value; (ii) plans for, including timing, development and progress of commercialization or regulatory timelines for our products, including NUPLAZID and DAYBUE, and our product candidates; (iii) benefits to be derived from and efficacy of our products, including the potential advantages of our products and product candidates; (iv) the timing and conduct of and topline results for our clinical trials, including the timing of topline results from our clinical trial in Prader-Willi syndrome, the continuing enrollment in our clinical trials in Alzheimer’s disease psychosis, the initiation of our clinical trial in Lewy Body Dementia Psychosis, and the timing and content of our presentations or announcements regarding our clinical trials; (v) our estimates regarding our future financial performance, profitability, capital requirements or expenses, including estimates of potential commercial product and pipeline peak sales and potential net sales of our commercial franchises in 2025, and (vi) our ability to successfully complete additional business development transactions. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to: our dependency on the continued successful commercialization of our products and our ability to maintain or increase sales of our products; our plans to commercialize DAYBUE globally and trofinetide in the EU; the costs of our commercialization plans and development programs, and the financial impact or revenues from any commercialization we undertake; our ability to obtain necessary regulatory approvals for our product candidates and, if and when approved, market acceptance of our products; the risks inherent with product candidate development, including risks of unsuccessful clinical trial enrollment and negative or inconsistent results; our dependence on third-party collaborators, clinical research organizations, manufacturers, suppliers and distributors; the impact of competitive products and therapies; our ability to generate or obtain the necessary capital to fund our operations; our ability to grow, equip and train our specialized sales forces; our ability to manage the growth and complexity of our organization; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2024 as well as our subsequent filings with the Securities and Exchange Commission from time to time, including our quarterly report on Form 10-Q for the quarter ended March 31, 2025. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250625948610/en/